Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...

Learning life skills to handle Duchenne muscular dystrophy can include health management, working with caregivers, and ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

Duchenne muscular dystrophy (DMD) is the most common genetic muscle disorder, affecting 23,000 boys in the US each year. The disease results from a mutation in the gene that encodes dystrophin — the ...

"Epicrispr secures $68m for facioscapulohumeral muscular dystrophy therapy trial" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

Get Instant Summarized Text (Gist) Gene therapy for Duchenne muscular dystrophy (DMD) faces challenges as the immune system may hinder its effectiveness. A large-scale trial showed that the ...